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Changing society and its views on disability, one column at a time
Like me, my colleagues here at Bionews, the parent company of this website, write about […]
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EMA agency continues to oppose renewing Translarna’s approval
The Committee for Medicinal Products for Human Use (CHMP) — part of the European Medicines […]
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I wish all of my sons could have played football
I sat in the front row of the football stadium, watching my second-oldest son, Chance, […]
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CIFFREO Data Discussion Video
Watch the video HERE Pfizer recently provided this video updating the results from their Phase […]
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An emergency preparedness plan is crucial for those with disabilities
As Western Pennsylvania natives, my wife, Wendy, and I are both Steelers fans. Naturally, when […]
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RNA-based approach shows promise for DM1 in cell models
Treatment with antimiRs, an RNA-based therapy approach, was able to correct molecular defects in a […]
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Gene therapy at low dose helps two OPMD patients with swallowing
A single low dose of the investigational gene therapy BB-301 helped with swallowing for the […]
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Catch Up on World Muscle Society 2024
Each year, the World Muscle Society (WMS) Annual Congress brings together the best minds in […]
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WATCH: SHIELD DMD – A Bone Health Study in Duchenne (Webinar Recording)
Bone health is an important aspect of living with Duchenne muscular dystrophy. Roche recently joined […]
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Capricor now seeking FDA approval of DMD therapy deramiocel
Capricor Therapeutics has started its rolling submission seeking U.S. approval of deramiocel, a cell therapy […]
